Background: Although small-cell lung cancer (SCLC) is a primarily high
ly chemosensitive malignancy, the majority of patients will ultimately
relapse. As part of an ongoing effort to identify active new agents,
the efficacy of treosulfan in patients with relapsed SCLC was examined
in a monoinstitutional, phase II trial. Material and Methods: Between
January 1995 and September 1996, 15 previously treated patients (limi
ted; disease n = 1, extensive disease n = 14) with refractory (n = 1)
or relapsed (n = 14) SCLC were included in this prospective phase II s
tudy. The patients received treosulfan 8 g/m(2) intravenously as a sho
rt infusion every 4 weeks. The application was terminated in case of d
isease progression. Results: 14 patients are evaluable for response an
d toxicity after receiving 1-6 courses of treosulfan. No complete or p
artial remissions were seen. Three patients achieved a stable disease
lasting 2, 3 and 10 months, respectively. Eleven patients showed disea
se progression. The most common toxicities were thrombocytopenia (14%
WHO grade 3-4) and leukocytopenia (5% WHO grade 3). Nonhematological t
oxicities, e.g. alopecia, nausea, vomiting, stomatitis and skin reacti
ons, were moderate and mild. Conclusions: No major efficacy was seen u
sing treosulfan as second-line therapy in patients with extensive dise
ase SCLC. There are no sufficient data, however, on the effectiveness
of treosulfan in front-line therapy.